PTC Therapeutics, Inc. (PTCT) Q2 2017 Earnings Report, Transcript and Summary

Good day, ladies and gentlemen, and welcome to the PTC Therapeutics Second Quarter Financial Results Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference is being recorded. I’d like to introduce your host for today’s conference Emily Hill. Ma'am, please begin.

Good afternoon, and thank you for joining us to discuss our 2017 second quarter corporate updates and financial results. Before I hand the call over to our CEO, Stuart Peltz, I would do remind you that today will be making forward-looking statements. These statements include all statements other than those of historical effects, including statements concerning financial guidance, our expectations with respect to future commercial availability and access to EMFLAZA, the anticipated benefits of the EMFLAZA acquisition, our future expectations providing other clinical, regulatory and commercialization matters, including with respect to potential outcomes anticipated timelines, anticipated milestones and timelines of our SMA collaboration with Roche, addressable patient population for Translarna and EMFLAZA, and the potential success of Translarna for the treatment of nmDMD and EMFLAZA for the treatment of DMD. Actual results may differ materially from expressed or implied by forward-looking statements as a result of a variety of risks and uncertainties including those related to our continued commercial launch of EMFLAZA, including our ability to secure adequate pricing, coverage and reimbursement terms of third-party payers for other products in a timely manner, whether and to what extent third-party payers impose additional requirements before recruiting EMFLAZA prescription reimbursement. Our ability to integrate EMFLAZA into our business and realize the anticipated benefits of EMFLAZA acquisition changes in margin regulation, our ability to resolve the matter set forth in the future file will be received in FDA in connection with our NDA for Translarna for the treatment of nmDMD, including whether our filing of the NDA, proposed with FDA will result in timely successful review of the NDA. And those risks discussed under heading special note regarding forward-looking statements and Risk Factors in our second quarter Form 10-Q, which is available from the SEC and on our website. Such statements represent our judgment as of today and PTC undertakes no obligation to publicly update any forward-looking statements except as required by law. We will disclose certain non-GAAP information during this call. Information regarding use of GAAP and non-GAAP financial measures and a reconciliation of GAAP to non-GAAP is available on today's earnings release. With that, let me pass the call over to Stu.

Thanks, Emily. And thanks for joining us this afternoon. We are thrilled to report on the success of our business across multiple areas. This quarter, there has been significant advancement standing our commercial, clinical, regulatory and research programs. In particular, we will review the commercial progress we have made with our 2 products, EMFLAZA and Translarna, as well as the regulatory process for our Translarna application in the United States. I will also share advancements we've made in our spinal muscular atrophy program, including meaningful data recently presented at 2017 CureSMA meeting. Let me start with a commercial update. In mid-May, we've launched EMFLAZA in the United States for all Duchenne muscular dystrophy patients, age 5 years and older. As we said before, we acquired EMFLAZA in part because we understood from the placebo arm of our Duchenne clinical trials and the results from other industry and natural history studies, the strong clinical benefit EMFLAZA provides for Duchenne patients. We launched EMFLAZA with the intention of making this crucial therapy available for all eligible United States Duchenne patients. We are following through on this commitment and are happy to report that only 12 weeks into the launch, there are already over 1,200 patients on EMFLAZA. Our goal is to ensure broad access to all eligible patients. To that end, we have worked hard to minimize out-of-pocket cost for patients. To date, the monthly out-of-pocket burden for eligible patients has been close to 0. Duchenne treatment guidelines recommend corticosteroids as a foundational component of the standard of care. EMFLAZA is the only corticosteroids approved for Duchenne muscular dystrophy in the United States. We believe EMFLAZA should be prescribed to all eligible patients. And we are working towards this goal of broad access. We continue to see a positive response from patients and providers. We have also seen support from the payers, resulting in positive reimbursement sufficient. We are pleased the conversion of bridge to commercial have been faster than we originally expected. Though still early in the large, based on the progress to date, we are raising our 2017 EMFLAZA guidance from $15 million to $20 million. We will continue working diligently to make sure EMFLAZA's clinical benefits that we observed in our clinical trial and other trial is understood by patients and providers. Our investments in finding treatments for Duchenne muscular dystrophy started almost 20 years ago, focusing on RNA biology to treat disease-specific mutations. These research and development efforts resulted in what is now Translarna. Translarna enhances the rises of a Nonsense Mutation to allow the production of full length dystrophy. Translarna is currently commercially available in over 25 countries worldwide. In the 3 years since launch, we have seen tremendous uptake in the European Union and Latin America. And most recently, we have begun expanding into the Middle East and Central and Eastern Europe. Patients and physicians have expressed benefits from the therapy and compliance that they remain over 90%. We are pleased to report that strongest quarter for Translarna since launch 3 years ago, with revenue of approximately $46 million. At this point, we are increasing Translarna 2017 guidance to $120 million to $140 million. We are working diligently to bring this important therapy to nonsense mutation Duchenne muscular dystrophy patients in the United States. Translarna has a PDUFA date of October 24, and the FDA has tentatively scheduled our Advisory Committee meeting for September 28. We are eager for the opportunity to present the evidence supporting Translarna's benefits. We also look forward to hearing the perspective of key stakeholders, including Duchenne patients and their family. Some of whom have had access to this therapy since our first trial over 10 years ago. We are actively preparing for a potential United States launch. Our commercialization of EMFLAZA allowed us to establish an infrastructure in the United States, which we can leverage to quickly launch Translarna. Let me now switch gears and touch on our SMA program, a joint development collaboration with Roche and the SMA Foundation. Let me remind you, we have an SMN2 modifier, RG 7960. This quarter, we reported important clinical results from an interim analysis of the data from the SUNFISH study in type 2 and 3 SMA patients. We anticipate that this study will transition into a pivotal phase in the coming months. In additional trial, FIREFISH is evaluating RG 7916 is type 1 SMA patients. This study is also expected to transition to the pivotal stage later this year. As an oral small molecule with broad tissue distribution, RG 7916 has the potential for important clinical benefits and commercial differentiation. We will share more with you on the results later in the call. Our pipeline also continues to progress with additional indications for Translarna and programs in our [indiscernible] and oncology platform. Lastly, we are proud to be in a strong financial position. As you will see, the revenue generated from a commercial aspect, Translarna and EMFLAZA, greatly offsets our cash burden and moves us towards operating as a sustainable business. We will walk through these programs starting with a detailed commercial review from Mark Rothera. Mark?

Thanks, Stu. I'll focus first on Translarna. Our therapy approved by the European Medicines Agency for Nonsense Mutation Duchenne muscular dystrophy. Translarna is available in over 25 countries worldwide, including Europe, Latin America. And more recently, we have been expanding the Middle East and Central and Eastern European regions. In these countries, Translarna is obtained through either commercial access or reimbursed early access program. As Stu mentioned, this was our strongest quarter for Translarna sales since launch. We generated net revenues of approximately $46 million, which represent nearly 200% growth versus the same quarter last year. Sales from Latin America contributed to the strong growth this quarter. As we said before, some of this contribution includes large orders from Latin America that can come at irregular intervals. Overall, we have seen in good growth in all regions we operate in. As stated before, growth in Translarna comes from both increasing penetration in existing territories as well as the addition of new geographies in Central, Eastern Europe and the Middle East. We're working through final pricing and reimbursement negotiations in select countries. Notably, Translarna has been evaluated by Germany's Benefit Assessment twice and both times received a positive rating. As we have discussed multiple times, we have been successful with an alternative implementation pathway, which has allowed commercial access for both new and existing patients. As a matter of process, we will engage in another around the price arbitration. We will have the choice to remain on importation part, regardless of the outcome. We expect the arbitration process to be completed this quarter. In France, we have completed the Health Technology Assessment review and received a rating of ASMR 5. We are moving to pricing and reimbursement discussions with the French authorities and expect these negotiations to conclude no earlier than by the end of this year. Based on the overall dynamics of the business, we are in a position to raise Translarna's 2017 guidance, as Stu mentioned earlier. Now I will shift to the launch of EMFLAZA, which is for all eligible United States Duchenne muscular dystrophy patients, 5 years or older, irrespective of their mutation. The launch of EMFLAZA, which started in mid-May, has been a great success. This is in part because of our 20-year commitment in heritage to DMD as well as the deep commercial experience of our team. Working with patient groups, physicians and payers to ensure broad access to EMFLAZA continues to be our priority. I am happy to report that more than 1,200 patients in the U.S. are now receiving EMFLAZA through commercial or bridging programs just 12 weeks post launch. We have presented placebo data from the ACT DMD trial at the International meeting in May, which shows that patients from EMFLAZA had a benefit in muscle function, which correlated with up to 40 years in the later loss of ambulation. This data was part of the motivation for the EMFLAZA acquisition. And we are proud to bring this therapy to all eligible U.S. patients, most of whom previously did not have access. Specifically on access, coverage has been strong. To date, 47 states are covering EMFLAZA in Medicaid program and over 170 private insurers have authorized EMFLAZA reimbursement. Collectively, over $250 million lives are covered by these private and public insurance programs. Importantly, the average monthly out-of-pocket cost for EMFLAZA to patients is close to 0. We'll continue to provide patient numbers for the remainder of this year as a useful metric to track the launch. Beyond 2017, we will solely provide revenue guidance. As Stu mentioned earlier, we are increasing our 2017 revenue guidance for EMFLAZA from $15 million to $20 million, from $5 million to $10 million. This is a reflection of the early success of the launch and our execution in bringing access to patients broadly. We continue to convert prescriptions and see solid growth ahead. As we progress through the launch, the next phase will include the challenge of educating physicians and patients to broaden the use of EMFLAZA to those who are naive to corticosteroid therapy. Based on the efficacy and safety profile, we believe EMFLAZA should be standard of care for all eligible patients. The most important aspects of the message we want to share with key stakeholders is the efficacy benefit seen with EMFLAZA. As mentioned earlier, the data from our ACT DMD trial shows that patients from EMFLAZA had a benefit in muscle function, which correlated with up to 4 years in delay to loss of ambulation. Additional datasets will be published this year and are important for key stakeholders to appreciate the magnitude of the benefit from this therapy. I am very proud of what we've built here at PTC over the last 4 years. We are now a global commercial organization with a highly talented team in the orphan space, representing 2 of the 3 approved DMD-specific therapies in the world. And I would like to turn it back to Stu.

Thanks, Mark. Before we move on from the commercial update, I wanted to take a moment and thank, Mark, for his hard work and effort over the past 4.5 years. Mark has moving on in his career. And I want to recognize the tremendous achievement of building our global commercial enterprise and successfully positioning us as a sustainable commercial business. Over 20 years, PTC has evolved from a small New Jersey R&D focused biotech to a global commercial stage company with continued R&D innovation. As you are aware, we recently restructured and I am now positioned to maximize our commercial clinical and research assets going forward. Let me now provide a few additional updates on the development program for Translarna. We have 2 ongoing studies for Duchenne muscular dystrophy. The first is the pediatric PK and safety study of Translarna in children ages 2 to 5 years old, which will be completed this year. Our intention is to file for an extended label in this population. Our long-term study in Translarna opened to start enrolling patients this quarter. As a reminder, this trial is a specific obligation of our EMA approval. We're also excited about our ongoing Phase II Translarna studies in both and we'll be highlighting these Phase II clinical trials at our upcoming Analyst Day later this year. Let me now switch to another one of our programs, which is based on our small molecule platform. This technology has been used to discover potential new therapies for spinal muscular atrophy, or SMA, a rare generic neuromuscular disorder that generally manifest earlier in life and is the leading genetic cause of death in infants and toddlers. We have a robust program collaboration with Roche and SMA Foundation around oral SMAs with modifiers. Spinal muscular atrophy is caused by reduced level of SMA proteins due to deletion and mutation of the SMA 1G. The oral compound RG7916, we had a clinical development is designed to drive alternative slightly of the mRNA to produce increased levels of full-length mRNA and functional SMN2 routine. We believe that an oral therapy with exposure throughout the body, which includes both peripheral tissues such as muscle, liver, bones and nerve tissue will be more beneficial than those focused on just the central nervous system. The least compound RG7916 is currently being studied in 3 clinical trials in SMA patients. SUNFISH is a double-blinded placebo-controlled randomized study. The first part has enrolled approximately 50 SMA type 2 and 3 patients. The second pivotal phase of the study is planned to enroll a 168 patients with a 12-month end point of motor function measure. Interim data analysis from the dose finding phase of SUNFISH was recently presented at the CureSMA meeting. The interim data demonstrated an approximately 400% increase in the SMN2 full-length, the Delta 7 mRNA ratio in a dose-dependent manner. A linear relationship was observed between media exposure levels and increase in the ratio of full-length adult 7 SMN2 investments in RNA. For reference, RG1700, our first compound, demonstrated an approximate average increase of 100% in SMN2 mRNA, which translated to an average 50% full length SMN protein. The interim analysis also noted that while the first patients has been on therapy for 6 months. No toxicities requiring patients withdrawal has been observed in the clinic. Additional data from part 1 of SUNFISH, including protein levels, will be presented at a major medical meeting later this year. The SUNFISH study is expected to transition through a pivotal phase in the coming months. FIREFISH is an open label 2-part study in SMA part 1 in-patient. The current ongoing dose escalation phase of the study is enrolling up to 10 patients. The study will then transition to 40-patient pivotal trial. The primary endpoint is sitting without support as assessed in the daily growth motor scale. The geographical areas or other therapies are not yet available. Prior is also expected to transition to a pivotal portion later this year. The SMA program also has an open-label trial known as Gelfish [ph], Gelfish allows SMA patients from other studies SMN2 slightly targeting therapies, including our previous candidate RG7800 to roll over to RG7916. This trial was developed out of request from patients and their families for continued access to our oral slicing therapy. The collaborative work ongoing with Roche and the SMA Foundation demonstrates the productivity of what slicing technology. Outside of this collaboration, we are also pursuing additional preclinical programs, targeting earnings slightly. Exciting work in both Huntington's, the familial dysautonomia is ongoing. At an Analyst Day later this year, we will share more about these slicing programs as well as highlighting our R&D pipeline for next-generation read-through program, our oncology program and our clinical development program for Translarna and other indications. I would like to now turn the call over to Christine Utter, our Principal Financial Officer. Christine?

Thanks, Steve. I am pleased with the strong performance this quarter. We reported our first EMFLAZA revenues and achieved our highest quarterly Translarna revenues to date. Translarna revenue in the quarter was $45.8 million, a 73% increase over the prior quarter. As Mark noted earlier, starting large orders from Latin America contributed to this notably high quarter Translarna revenue. EMFLAZA revenue was reported at $2.1 million. The early launch of EMFLAZA has been a successful process. The performance of both of these commercial products allows us to increase our 2017 revenue guidance, which I will detail shortly. I now like to review our financial details for the second quarter of 2017. We reported a net loss of approximately $17.5 million for the second quarter of 2017, which declined over $21.4 million from a $38.9 million loss for the same period of 2016. Our operating losses are anticipated to continue declining as we leverage our existing infrastructure and continue to grow our revenue base now with 2 commercial products. GAAP R&D expenses were approximately $30.8 million for the second quarter of 2017 compared to approximately $28.8 million for the same period in 2016. Non-GAAP R&D expenses in the second quarter were approximately $26.9 million compared to $24.6 million for the same period in 2016, representing a year-over-year increase of $2.3 million. R&D expenses have increased as a result of the initiation of long-term DMD studies and increased laboratory cost, partially offset by expenses associated with the completion of our CF program. GAAP SG&A expenses were approximately $28.9 million for the second quarter of 2017 compared to approximately $23.4 million for the same period in 2016. Non-GAAP SG&A expenses were approximately $24.9 million for the second quarter of 2017 compared to approximately $18.3 million for the same period in 2016, an increase of $6.6 million. SG&A expenses had increased mostly driven by the launch of EMFLAZA. We would like to take this time today to review our 2017 revenue and operating expense guidance. We are updating our Translarna net revenue guidance to $120 million to $140 million, up from $115 million to $130 million. As we noted, Translarna revenue remains strong with growth continuing in both new and existing territories. This guidance assumes the current exchange rate and a continued commercial expansion for Translarna and nonsense mutation DMD outside of the U.S. We are also raising EMFLAZA guidance to $15 million to $20 million, up from $5 million to $10 million to reflect the early success of the launch. We also anticipate a $20 million milestone payment related to the SMA program in the second half of the year, for potential totaled 2017 revenues of $155 million to $180 million. We are reiterating our GAAP operating expense guidance for the year 2017 between $250 million to $260 million. Excluding estimated non-cash stock-based compensation expense of approximately $40 million, full year 2017 non-GAAP operating expenses are anticipated to be between $210 million and $220 million. These expenses are expected to be primarily in support of the continued research and clinical development of our product pipeline candidates as well as a commercialization of Translarna outside of the U.S. and investing in the commercial launch of EMFLAZA in the U.S. Our previous cash guidance was to complete 2017 with approximately $100 million. Due to decreased cash burn, we now anticipate ending the year with over $120 million of cash and marketable securities. We're happy to end this quarter in an even stronger financial position. Let me hand the call to Stuart.

Thanks, Christine. It's been a great quarter. Before we hand over the call for Q&A, I wanted to let everyone know, considering the FDA's ongoing review of our Translarna NDA and upcoming in PDUFA, we will not be making further comments beyond the prepared remarks on this laboratory process. Okay, operator, you can open the line for Q&A.

[Operator Instructions] Our first question is from Alethia Young of Credit Suisse. Your line is open.

Thanks for taking my question. This is Derek on for Alethia. So congrats for a strong quarter. I have one question. So beyond the 12,000 [ph] patients you have, I'm wondering how many patients like [indiscernible] but still waiting and how many percentage of patients got lied by the payer, and any kind of like push backs on the pay - on the pricing side will be helpful? Thank you.

Thanks, Derek. Thanks for the question. As we said, yes, things have been moving where we currently have 1,200 patients either on insurance or on bridging. I should make the point that all those patients have insurance, since we anticipate they all ultimately turn into commercial payers. And while we don't give precise numbers and we don't give numbers disclosed on the prescriptions, I think the fact that we have increased our guidance from 5 to 10 to 15 to 20, I think, it is a reflection of that. And I think in the sense of consequence really of just how fast conversions are going. So we feel good about it and we think that is still a pipeline of outpatients to go through the process.

Thank you. Our next question is from Matthew Eckler of RBC Capital. Your line is open.

Yes. Hi, thanks for taking the question. So I wondered if you comment a little bit about the average age and average weights of the 1,200 patients currently on EMFLAZA as well as what trends you've seen so far and then how you think that could change going forward?

Yes, sure. Thanks, Matt, for the question. So just to remind everyone, we previously disclosed that we anticipate that for 25 - typical patients about 25 kilograms and then that price would be $35,000. And that's really what we're disclosing. At this point, we're not disclosing additional average rates. What we did at the 25-kilogram typical patient is what we ultimately expect in terms of the prescribing patents for DMD. So at this point, it's actually probably still too early in the launch to see where ultimately that would go.

Okay. Okay, great. Thanks. And then could you talk a little bit about what you're seeing in terms of the prior authorization requirements for EMFLAZA?

Yes, sure. So in terms prior authorization what we expect -- first of all, I should say that reimbursement coverage is obviously going pretty well in that. It's reflected in the increasing in guidance that we did. We anticipate or have seen predominantly in prior authorization step added previously. And so -- but we've been able to either get through that relatively rapidly, so that while there we haven't seen yes that to be a major problem this far. So these patients who have been on -- many of them who have been on the - are actually getting rapidly through the conversion process.

Okay, great. And then, just one more quick one here. You mentioned there will be additional EMFLAZA data later this year. Could you provide any more detail on what that data is and when we might see it?

Yes. So as you may recall, we had a poster of our results -- of the top line results from our trial from the placebo group, which looked at the patients that who are placebo, but who are on versus the in that poster described the benefits that we saw over EMFLAZA. I think, the benefits of EMFLAZA over prednisone. And so, I think, what you will see is there will be additional work. I know there is a publication that will be coming out from the synergy natural history study, looking at the natural history and the positive effects of corticosteroids in general, but also calling out the comparisons of EMFLAZA versus prednisone. And again, you will see the benefits of EMFLAZA over prednisone within that publication as well. And then, there will be some discussions, I think, and work at some point and some of the data, which also has a placebo, which also has prednisone and EMFLAZA. And you'll see the consistency of the results of where EMFLAZA patients did better than prednisone patients there as well. And I think, we'll have something where we'll use not only our data, but also combine the data in the meta analysis. So I think, at the end of the day what you're going to see is a fair number - a fair amount of evidence that is showing the benefits of EMFLAZA, and why we think it should be ultimately the standard of care for these patients.

Okay, great. Thanks a lot for taking the questions.

Thanks, Matt.

Thank you. Our next question is from Paul Tray [ph] of Barclays. Your line is open.

Hi. Thank you. Good afternoon and thanks for taking our questions. I guess, my first question is on SMA. And you mentioned that you would have SUNFISH data later this year. And with regard to the transition to a potential pivotal trial here, can you comment on how you're thinking about the primary biomarker endpoint versus your potential secondary functional endpoints and will this transition to the pivotal trial include a sort of meaningful motor milestones as well as endpoints?

Yes, thanks for that. So just -- again, just to remind everybody. We had seen -- we had Roche and SMA Foundation build a nice poster show of about 400 increase in the amount of SMA and full-length transcript versus the SMN2 Delta RNA. And so there was a significant increase. Just for comparison if you look to what we had in RG1800 before, we had 100% increase of the full-length transcript and about 50% increase of protein level that was the average amount. So you're seeing a substantial increase with RG7916. This is what we reported as the SMA meeting was RNA level. Later this year, we'll be talking about the protein levels and that will be a meeting hopefully this year as well. And then, what we will do is transition from it was an escalating dose phase, but then transitioned into a pivotal phase. And the purpose there is, obviously, to go -- to have the biomarker, which will continue to measure. And again, we're able to because it's a systematic drug itself, it is a systematic drug and it has exposure to a tissue, that's why we can look in the blood and see increased levels both in RNA and protein. So that's the advantage of that. And then, when you transition, for instance, in the FIREFISH and your SUNFISH study, that's a 12-month study, but uses a motor function, the MFM scale, to measure a 12-month of treatment. And therefore, we'll look at within that, various measurements within that trial. And also, then we're able to calibrate that to the levels of SMA protein. The FIREFISH is type 2 and 3 patients. In the type 1 patients, we have that's for type 1 patients. And there we're using the daily infant scale, which basically looks also at sitting on a 5 to 12 months. So those are the clinical benefits outputs that we'll be looking at.

Great. And then just as a follow-up to the potential trial design there. How are you thinking about age ratification potentially within the type 2 and type 3 population. Is there any sense that there would be any better response with earlier or younger patients? And how we should potentially think about them that as a backdoor in your trial and potential outcomes.

Yes, so basically, I think we're doing some stratification in the type 2, 3 patients are 2 to 11 and 12 to 25 in terms of the stratification that are there. And those information, I think, you could see as the clinical trial for details of the trials

Okay. Great. Thanks for taking our questions.

Sure. Thank you.

Thank you. Our next question is from on Anupam Rama of JPMorgan. Your line is open.

Hey, guys. It's Eric. Thanks for taking the questions. Just a couple ones from last - just wondered if you could talk little bit about the concentration of the prescriber base. Here to start how we should be thinking about sort of the pace of patient booking beyond this initial bolus. And then, I know, Stu, you had comment on sort of average patient weight and age at this point. But I'm just wondering, if with regard to kind of net pressure going forward, whether there any anticipated sensitivity as it relates to sort of discounting given your experience this far on the commercial setting? Thanks.

Yes. So that's a good question. In terms of the physicians, I think it's been widely used around the country. I don't think there's any net in one area versus the other. I think that is used around the country. I think the way we think in general about, this is obviously there is pent-up demand. And we said prior to the launch, we have bolus of about 900 scripts that were ready to actually get through the process. And so that has been ongoing. So there is obviously a fair amount of progress that we've made into that. We are finding also on top of having EMFLAZA patients. There are also some naive patients. But you can imagine that ramp or that bolus that we have had recently are those that have been on EMFLAZA rapidly transitioning into EMFLAZA commercial EMFLAZA. And so that's being going about. We think there -- we do see some of the naive patients also getting through. But I think the next phase of what will be doing is to get patients who haven't been on any corticosteroid before that is to get through this process. And we imagine that would take more time than what we have seen here. So we still think there is growth that will occur and think the transition to a change that slope as it takes time to get patients on from naive to EMFLAZA. And talking to physicians, explaining the data that we have in others are putting on. So they understand that we think ultimately our goal is to make EMFLAZA the standard of care for these patients. Did I get everything

The second follow-up is on net pricing and just sort of sensitivity to the initial pricing assumption or net pricing assumption, given your level of sort of discounting or buying down that you currently experienced?

Sure. So obviously, what we've said is we're looking at 35,000, 25 kilos what we've disclosed. Our goal obviously is to provide access for all patients that we're working towards that. And as I said in my prepared remarks that really the co-pay and other. We have very strong programs. And therefore, our goal was to make sure that patients either wasn't out-of-pocket expensive than we've seen thus far really low to the minimums levels of out-of-pocket. So that's actually gone well. So that's where we are right now in terms of our understanding co-pay. That's been going while the program has been doing well. And what we're disclosing is the 25-kilogram patients $35,000.

Got it. That’s very helpful. Thanks for taking the questions.

Okay. Thanks, Eric.

Thank you. Our next question is from Alexandria [ph] of Cowen. Your line is open.

Hi. Guys. Thanks for taking my question. Just I’ll start with one on Translarna. Can you give us any more detail on the relative contributions of revenues from Latin America? And are there any particular quarters you would expect useful quarters?

To be what? For 4 quarters, yes. Thanks for that question. So obviously, we haven't disclosed overall revenue. We think, in general, it's actually going quite well, which is why we raised the guidance to 1 20 to 1 40. Latin America has been growing quite well, but we -- but there is not only with Brazil and other countries, there are a regular orders that go on. So there will be bulky -- there will times like -- there will be lumpiness in terms of orders. So it is going well. But I should point out really that all regions and part of why we raised revenue. All regions are still actually growing revenues.

Got it. And then just one on EMFLAZA. You've previously mentioned doing essentially expansion into younger patients. Can you give any more detail on your transfer and when we expect the trial?

Yes, sure. We can label as 5 and above. There is a plan to go into patients under 5, through the 5 -- to get to 2 to 4 years old. And we're working through that now on moving forward that in terms of how best to do that. So that's in the plan now. And working up to the protocol.

Got it. Thank you.

Thank you. Our next question is from the Joel Beatty of Citigroup. Your line is open,

Hi. This is Shawn calling on for Joel. I appreciate you taking time for my questions. Regarding the 7916 compound. What duration of follow-up, and what data would be most important to give you confidence and the decision to add the program?

Yes. Thanks, Shawn, for that question. So actually already the results that were talking about, we're showing there was a 400% increase in terms of the RNA levels, and we'll have the levels that will report to our review some time this year. But that gives us -- we already feel that's going to give us great confidence. But we're really doing in the 2-part study is that in the first part of the study really to do an escalating dose space to find the highest dose that gives us the change in the slicing. From that it's a seamless transition into the pivotal trial phase, where we go on into the clinical benefit. So really the decision, it's not a go -- no go decision, the decision on which dose do we go to.

And as a quick follow-up. Are you able to give any enrollment updates on the SMA type 1one and study?

Yes. I think what we're saying is that the first parts of both studies will be completed this year. So in the coming months, there will be then transitioned into the pivotal study.

Thank you. At this time, there are no other questions in queue. I'll turn it to Mr. Stu for closing remarks.

Well, thank you. I think as you can see from this quarter, we're having an infection point, where we have had 2 commercial products and an innovative R&D engine. Our mission has always been to provide treatments to patients living with rare diseases, who have limited treatment options. I'm proud Translarna, the first therapy for muscular dystrophy patient and will remain dedicated to working with physicians, patients and their families to bring Translarna to the patients in the United States. We're also pleased with the successful early launch of EMFLAZA. Consistent with the mission, we're working to ensure broad access to all eligible EMFLAZA Duchenne muscular dystrophy patients. Thank you all for joining the call.

Ladies and gentlemen, thank you for your participation in today's conference. This concludes your program. You may now disconnect. Everyone, have a great day.